Another biopharma executive went before Congress to explain drug price hikes. Unlike Martin Shkreli, Mylan CEO Heather Bresch actually answered questions. Taking heat over the EpiPen scandal, she tried to stick to her script, getting testy when legislators brought up a USA Today story about her mother helping market the life-saving anti-allergy injection to schools. Elsewhere in Washington, the NIH chief warned about Congress’s inability to agree on Zika funding, and Vice President Joe Biden talked with Stat about cancer research.
But the biggest Beltway life-sciences story of the week was the Food and Drug Administration’s stunning approval of the drug eteplirsen (Exondys51). It feels odd to say it was the FDA’s approval, when in fact it was one top administrator’s decision, overruling many others… but read on. Let’s start with more on eteplirsen, and round up the rest of the week’s biggest news.
—In a controversial decision with potential consequences for the future regulation of medical treatments, the FDA gave accelerated approval to the first drug for Duchenne muscular dystrophy, eteplirsen, from Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT). Shares of Sarepta boomed and the Duchenne patient community cheered. But documents released by the FDA showed that eteplirsen was only approved after Janet Woodcock, the FDA’s top drug evaluator, went against the recommendations of outside experts and the agency’s own scientists and reviewers. Her colleagues were critical of the drug’s data and fearful of the precedent it might set for drug approvals and the power of patient lobbying. Sarepta said it expects to launch eterlipsen at an average annual price of $300,000 per patient, a figure not likely to be challenged by insurers.
—Great Britain’s GlaxoSmithKline (NYSE: GSK) became the first big drug firm to appoint a woman as CEO. Emma Walmsley will succeed Andrew Witty after running GSK’s consumer healthcare business for five years.
—Irish-domiciled Allergan (NYSE: AGN) kept up its year-long shopping spree, placing bets on drugs to treat the liver disease nonalcoholic steatohepatitis—a severe type of fatty liver brought on by obesity, in large part. It’s a growing problem, prompting Allergan to commit up to $1.7 billion for Bay Area biotech Tobira Therapeutics (NASDAQ: TBRA) despite shaky Phase 2 data for Tobira’s most advanced NASH drug. Allergan also bought Akarna Therapeutics, another NASH drug developer, for a much smaller sum.
—Facebook founder Mark Zuckerberg and his wife Priscilla Chan, a pediatrician, said the Zuckerberg-Chan Initiative, a holding company for their Facebook fortune, would invest $3 billion to “cure, prevent, or manage all diseases” by the end of the century. The first concrete step is $600 million to establish a “biohub” in San Francisco that will bring together researchers from Stanford University, and the University of California campuses in San Francisco and Berkeley.
—Bill Gates, who has turned his own tech wealth into the world’s richest charitable foundation, was in San Francisco to cheer on Zuckerberg and Chan. His former company Microsoft made its own grand health pronouncement this week: a computational-intensive push to “solve” cancer in ten years, leading one participant to predict a “century free of cancer.”
—Privately held Tarveda Therapeutics, of Watertown, MA, could pay up to $163 million to Madrigal Pharmaceuticals (NASDAQ: MDGL) to acquire the old cancer work of Synta Pharmaceuticals, which merged with Madrigal in April after Synta’s lead drug failed in clinical trials.
—Israel’s Teva Pharmaceutical (NYSE: TEVA) paid Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, $250 million upfront for partial rights to fasinumab, an experimental drug for chronic pain. Teva and Regeneron are chasing a rival drug from Pfizer and Eli Lilly. Both represent an alternative to addictive opioids but have been dogged by safety problems.
—Roche’s Genentech division is paying Germany’s BioNTech $310 million in upfront and near-term milestones to co-develop cancer vaccines.
—Trials and tribulations: Gilead Sciences (NASDAQ: GILD) of Foster City, CA, is ending a Phase 2/3 trial of an ulcerative colitis treatment because it isn’t helping patients… Mast Therapeutics (NYSEMKT: MSTX) announced its sickle cell treatment vepoloxamer failed a Phase 3 trial… Shire wants to dump its interest in pacritinib, a failed myelofibrosis drug from Seattle’s CTI Biopharma (NASDAQ: CTIC), that it inherited when it bought Baxalta this year.
—Following the failure of its lead eye-disease drug, Cambridge-based Eleven Biotherapeutics (NASDAQ: EBIO) acquired Toronto’s Viventia Bio in an all-stock deal and rebranded as a cancer drug developer.
—Biotech build-outs: Cambridge-based Moderna Therapeutics will spend $110 million to build a 200,000 square foot messenger RNA drug manufacturing facility in Norwood, MA, that should open by early 2018… Nearby Alnylam Pharmaceuticals (NASDAQ: ALNY) just opened a 100-employee commercial hub in the U.K.
—The Medicines Co. (NASDAQ: MDCO), of Parsippany, NJ, was awarded up to $132 million from the U.S. Biomedical Advanced Research and Development Authority to develop antibiotics to fight drug-resistant bacteria.
—Swiss Alzheimer’s drug developer AC Immune (NASDAQ: ACIU) raised $66 million before fees in its Nasdaq IPO… Durham, N.C.-based skin drug developer Novan (NASDAQ: NOVN) raised $45 million in its IPO. Xconomy’s Frank Vinluan profiled the company and its IPO plans last month.
Ben Fidler contributed to this report.
Photo of the U.S. Capitol courtesy of Kevin Dooley via Creative Commons.Reprints | Share:
UNDERWRITERS AND PARTNERS
My friend Eileen and I are planning to “phone-watch” the first presidential debate together on Monday. Just by calling her number in Louisiana from my couch in the Bay Area, I’ll hear hilarious commentary on the Clinton-Trump exchange from Eileen, a terrific reporter who’s wittier than most standup comics.
I found another name for this low-tech form of long distance TV show sharing in the New York Times: “sync-watching.” Beyond a simple phone call, far-flung friends are using any technical means possible—like Skype and live tweeting—to duplicate that cozy experience of sitting in front of the same TV together.
This is only one of the ways social media and other technologies are changing the experience of forming our views on election issues and choosing our candidates. Tech will probably reinforce our entrenched opinions on politics to some extent—but it may also expand our worldviews and ease access to the voting process. This year for the first time, virtual reality is part of the mix because VR headsets are now widely available.
A Bay Area virtual reality startup is about to add new dimensions to the sync-watching experience for viewers of NBC News’s coverage of events leading up to the presidential election on Nov 8.
AltspaceVR has created a virtual replica of NBC’s Democracy Plaza in New York’s Rockefeller Plaza, the backdrop for network anchors during the debates and on election night. Political junkies will be able to invite their friends to join them in Virtual Democracy Plaza for NBC’s four virtual debate watch parties, political comedy shows, and the vote count drama the night of the election.
Viewers can check out the virtual venue even if they don’t have VR headsets, by going to altvr.com/nbcnews via their PC or Mac. But headset users who join AltspaceVR will have some interesting social options.
If they don’t want to attend a debate-watching party with people of a different political stripe, they can sign up to share it only with their like-minded pals (appearing as avatars, but audible in their own voices.) AltspaceVR can clone the venues it creates—including Virtual Democracy Plaza—so users can choose to inhabit that venue with a bipartisan crowd or with only a single friend if they like. (See how it works here.)
Redwood City, CA-based AltspaceVR is just one of the tech companies that’s trying to draw in more users or customers by creating new projects that put an election spin on their core offerings.
MediaScience, a market research company based in Austin, TX, tries to detect hidden attitudes among members of test panels by capturing their facial expressions, eye movements, heart rate, and other biometric characteristics. MediaScience has mainly worked for companies such as Disney and ESPN, but this year it’s been inviting its panelists to watch videos of Clinton and Trump, the Austin American-Statesman reported. The use of biological metrics and neuroscience in marketing is starting to expand to political research and campaign strategy-making.
Educational technology company Newsela, which creates multiple versions of news articles geared to varying student reading levels, is recruiting teachers to get kids interested in reading election stories by giving them the chance to vote for their favorite candidates in Newsela’s unofficial student polls. Voting opens for the school kids on Oct. 17 and closes Nov. 1, a week before the grownups vote. Newsela plans to release the results of the student election on Nov. 2 so the students can talk about them in class or with their parents. Then they can compare their generation’s verdict with the outcome of the adult vote on election night.
New York-based Newsela aims to offer youngsters straight news without a partisan spin. But if adults want to immerse themselves in the battle of opinions, there’s a new app on the market.
Menlo Park, CA-based Aleya Labs timed the launch this month of its first consumer app, political news and commentary site Contempo, so it would debut in the final weeks before the presidential election. Aleya says Contempo presents a selection of news and opinion items culled from multiple sources on both sides of the political spectrum. The items are chosen based on their social media rankings among “political influencers” from both the conservative and liberal camps. These influencers include bloggers, columnists, think tanks, and satirists.
Users can read articles from both political factions if they want to break out of their personal filter bubbles. But they can also choose only the items that dovetail with their existing views—literally by swiping to the left or to the right. For further convenience, the stories are actually labeled in red or blue.
On another front, technology is changing the way we influence the polls that try to predict the likely outcomes of elections. News stories on the fluctuating leanings of the electorate used to cite surveys by traditional pollsters such as Gallup, which routinely relied on phone calls to landlines. But that method was losing reliability due to lower response rates, as well as the movement toward mobile phones.
Election poll stories now often cite online surveys conducted by companies such as Palo Alto, CA-based SurveyMonkey, which is collaborating with NBC News. For example, their poll this week showed Democratic presidential candidate Hillary Clinton regaining … Next Page »Reprints | Share:
Biotech CEO Jonathan Lim co-founded Ignyta in 2011 to commercialize a new approach to diagnosing and treating rheumatoid arthritis, based on discoveries that showed how changes in methyl group molecules associated with the human genome could change the way genes function.
Instead, Lim may end up showing how to breathe new life into a life sciences company that was flat-lining, at least in terms of developing new technology.
As a tool for diagnosing rheumatoid arthritis and other autoimmune diseases, Ignyta’s technology failed in early 2013, Lim said in a recent interview.
Lim (pictured above) was lucky in some respects, though. He had closed on $6 million in financing just a few months earlier. Of the options he faced at the time—forge ahead with the diagnostic tool despite adverse test results, close down the company and return the capital to investors, or come up with an alternative—Lim decided to pivot, and switched Ignyta’s focus from autoimmune diseases to cancer.
Now Lim is awaiting interim results of a global pivotal clinical trial that could determine Ignyta’s ultimate fate. He said he anticipates the company (NASDAQ: RXDX) will disclose its interim findings sometime next spring.
If the results of what Lim calls Startrk-2 confirms the data from a prior clinical trial, in which 24 patients with different solid tumors showed a 79 percent response rate, Ignyta would likely seek FDA approval for entrectinib, its lead drug candidate.
The outcome of the prior clinical trial (Startrk-1) was exciting, Lim said, because patients in the study were diagnosed with seven different tumor types, including non-small cell lung cancer, head and neck cancer, renal cell carcinoma, and melanoma. Despite the variety of tumor types, Ignyta’s genomic analyses showed that all of the patients enrolled in the study had one of five specific gene fusions. Such fusions occur when a piece of one chromosome fuses with part of another chromosome to form an oncogene, an abnormal gene capable of causing cancer.
Fusion oncogenes frequently act alone in driving cancers, Lim said, and the five specific oncogenes are part of … Next Page »Reprints | Share:
A core tenet of Xconomy—and the reason behind our broad coverage across information technology, life sciences, energy, and more—is that the most interesting and important innovations often arise from the intersection of different disciplines.
One of the most important intersections today is between agriculture, the life sciences, and information technology—as evidenced by the growing field of agtech. Increasingly, the knowledge built up in developing new drugs, studying the human genome, and other life science research is being brought to bear on the crops we grow—while the technologies we have developed to sense and monitor our homes and bodies are being applied to tracking our food, monitoring its storage conditions, nutritional components, and the like. And human food production is just one aspect of agricultural technology—a vast field that also includes animal food and more.
In short, the field is exploding. At Xconomy, we have seen growing investments in the sector—both in a rash of new agtech startups and by bigger players such as Monsanto, Bayer Crop Sciences, and Syngenta. The annual AgTech Investment Report from AgFunderNews found that investment in agriculture and food-related technology startups alone hit $4.6 billion last year, roughly double the 2014 total.
I am pleased to announce that as of today, Xconomy is enhancing and expanding its coverage of this sector by launching a national Agtech and Food channel. This channel of our website, with its own home page, will feature all articles about these subjects and related topics from across our 11-market network. It is our ninth such subject-specific channel, joining Startups, Health IT, Cleantech, Exome (life sciences), Xperience (consumer tech and society), Fintech, Robotics and A.I., and Cybersecurity.
We’d like to thank the launch sponsors/underwriters who have joined us to support this new channel: Alexandria Real Estate Equities, Michigan State University, the North Carolina Biotechnology Center, and Spensa Technologies.
Xconomy editors around our network will be contributing to the new channel. Today we have three articles to kick off our coverage. One features a map of agtech companies in North Carolina, which has emerged as arguably the nation’s leading agtech cluster. We’ve identified 32 representative companies to date—and the list is far from comprehensive (we plan to keep adding to it as we learn about more companies). A companion piece focuses on a new institute and sub-cluster of companies and research groups developing in the Raleigh-Durham area around soil health. This sub-field has big implications for farmers, business, and agtech as a whole.
Our third article covers new efforts in microfarming, which use the latest in sensors and smartphone apps to help people grow their own food.
Coming down the pike, we will be reporting on efforts in areas such as urban agriculture, water and sustainability, robotics, and other innovative approaches in farming and food.
With the launch of this channel, we make it easy for readers interested in agtech and food to find all articles on these subjects from around our network. All you have to do is go to the home page of the new channel, or sign up for a dedicated e-mail newsletter.
We hope our agtech coverage grows on you.Reprints | Share:
Amid the hue and cry over the Mylan EpiPen pricing debacle, Hillary Clinton unveiled her “Plan to Respond to Unjustified Price Hikes for Long-Available Drugs.” Clinton’s plan calls for “dedicated oversight to protect consumers.” To that end she would “convene representatives of federal agencies…to create a dedicated group charged with protecting consumers from outlier price increases. They would work closely with and be advised by patient advocates, independent non-government experts on drug pricing and comparative effectiveness, and state and local regulators to investigate and respond” to unjustified pricing activities.
Charley Grant, writing in The Wall Street Journal, commented that, “Any action to curb price increases on older drugs would hurt manufacturers that rely on them to generate growth.” But he reassured investors “that scenario is unlikely to come to pass.” Good advice for investors; not so good for industry.
That dependence on price increases won’t change any time soon. These ideas—if not the specifics—constitute a real threat the pharmaceutical industry must take seriously. The Democratic presidential nominee has proposed a nuclear solution for pricing generics that, once established, would almost certainly get out of hand, and could end the business of making proprietary drugs as we know it.
Clinton’s plan does not seem far-fetched in light of a possible Democratic sweep that could see appointments to high office for people like Democratic senators Elizabeth Warren and Bernie Sanders. Calling for cheap imports and Medicare price negotiations, Donald Trump, too, has demonized “Big Pharma.”
Grant pointed out that, “The harsher aspects, such as a panel to review the fairness of drug pricing or a change to Medicare negotiating policy, would likely need congressional approval.” However, in a government by executive order, such as we have today, many of the constitutional checks and balances like Congressional oversight don’t apply. Judicial redress, as the failed Pfizer-Allergan merger demonstrated, cannot provide relief in a commercially relevant timeframe.
Allergan CEO Brent Saunders recently told Reuters, “My biggest fear, every time there’s another headline…is that venture capital needed for innovation [and early drug discovery] could move out of the sector… . It has started.” In an article for the January edition of the MedicineMaker Markus Thunacke and Pauline Ceccato, made a similar point that with “the implementation of tighter pricing systems in both the US and EU…[w]e may end up in a situation where drug makers will shift their focus from risky, costly R&D to cheaper, more stable generics/biosimilar businesses…at a great cost for innovation and, ultimately, patients’ health.”
A number of industry pundits addressed the crisis in blog posts that condemned “irresponsible price gouging” and described possible remedies, including transparent pricing, increased competition through accelerated approval of generics and value-based pricing of proprietary drugs. The proposals from the politicians and the pharma experts reflect contradictory perspectives on an issue of existential importance to the industry. The future of the drug business may well depend on its ability to reconcile the two.
Interestingly, both sides drew the critical distinction between generic and proprietary drugs and the need for different pricing mechanisms. In his blog Bruce Booth of Atlas Ventures contrasted “exploiters and innovators,” the former a subset of the generic industry. However, as he points out, it would be wrong to place too much confidence in the public’s ability to distinguish between the two—controlled prices only for generics. John Lechleiter, CEO of Lilly, once summed up a day on Capitol Hill, by saying that he thought he might have convinced a few Congressmen that generic companies don’t invent generic drugs. From the perspective of voters and politicians, what is important is not whether a drug is generic or proprietary, but whether they need it.
Clinton proposes to combat “unjustified price hikes.” What constitutes “unjustified?” Is it like former Justice Potter Stewart’s definition of pornography, “I know it when I see it?” If so, it is a license to intercede whenever politically expedient, a standard that will not serve the industry well in the long run. Do we want to place politicians in the position of weighing profits for drug companies against the sanctity of human life? What is justified if health-care spending threatens to consume the Federal budget? Is any price “justified” when a child’s life is at stake?
Despite the outcry, as long as the industry continues to struggle with innovation, it will depend on price increases as a major driver of growth. Matt Herper at Forbes pointed out that the price of the world’s best-selling drug Humira increased at an average of 6% per quarter over the last three-and-a-half years and is expected to continue growing into the 2020s.
Even when innovation works, revolutionary treatment platforms can bring with them revolutionary price tags. Glybera, the first gene therapy drug approved in Europe, was introduced in Germany last year at 1.1 million Euros ($1.4 million). While this reflected the potential savings over a lifetime, the real-time impact on health-care budgets could be devastating.
Rather than accepting the question as Clinton has framed it, i.e. “What price is justified?” the industry must ask, “What is causing price dislocations and the failure of the market to accurately value goods and services?” In considering answers, it must be prepared to push beyond simplistic, emotionally-charged responses along the lines of “greedy CEOs” that address only the symptoms. Gretchen Morgenson devoted a column in The New York Times to executive pay at Mylan.
Competitive markets are the most-efficient mechanism yet devised for matching price and value. CEOs operate within the market landscape. Some may see a bit farther than others in setting prices, but the conditions that permit a 5,000-percent increase in the price of a generic drug represent a failure of the market. That should be the focus of inquiry, not what is a “justified” price.
In making its case to the public, the industry must concentrate on simple foundation concepts: efficient pricing of generics through market-driven competition, and value-based compensation for proprietary drugs—value in the avoided cost of previous treatments, in the quality and productivity of lives saved, in the incentive for future drugs.Reprints | Share:
Darin Andersen, the busy-bee creator of San Diego’s Cyberhive, iHive, and xHive startup programs, says eight early stage tech companies have joined the co-working space he has been building out in Bankers Hill under a new “Entrepreneur in Residence” program.
Andersen created the program under the auspices of CyberTech, a membership organization he created to provide networking, resources, and programs focused on cybersecurity, Internet of Things, and related technologies.
Earlier this month, the City of San Diego awarded a $40,000 grant to Nest, the 16,000-square-foot co-working space that Andersen founded, to help foster the creation of more startups and jobs in the region.
Under the six-month Entrepreneur in Residence program, startups teams pay a $140 monthly CyberTech membership for the first two months and $250 a month for the remaining four months, Andersen said. Enrolled companies may be focused on near-term goals that range from raising capital to recruiting key technical and business leaders. “Our goal is to get them on their feet from a sustainability standpoint,” Andersen said.
The participating startups are:
—SD3D: Automated 3D manufacturing.
—Secured Universe: Secure Android OS.
—Skylift Global: Heavy-lift drones.
—Sympathetic Innovations: 3D-printed, counter-IED training devices.
—TrueID Security: Biometric security protection.
—Breeze: Ride-sharing app with fuel-efficient cars.
—Object Security: Simplified security management.
—MiPOV: Wearable Internet of Things “techlace” (in stealth).Reprints | Share:
Think “innovation” and you might picture some scrappy entrepreneurs toiling away in a kitchen or garage, hoping the landlord doesn’t notice what they’re up to. But the reality is that turning science into big business often requires far more sophisticated infrastructure—and the landlord who provides that infrastructure sometimes has one of the best windows into the process.
For the life sciences industry, one of the largest landlords, and closest observers, is Alexandria Real Estate Equities. The Pasadena, CA-based urban office real estate investment trust (REIT), founded in 1994, has grown into one of the country’s largest developers of world-class collaborative science and technology campuses. Alexandria has put up buildings in virtually every key life science cluster in the U.S., from San Francisco, San Diego, and Seattle on the west coast to Boston, New York, and Research Triangle Park out east. Its campuses, often featuring dramatic lobbies, community organic gardens, conference and event centers, restaurants, and collaboration spaces, have in many cases become centerpieces of the local innovation ecosystem.
Lately, Alexandria is aggressively expanding its reach. It has recently announced two new startup platforms designed to support the growth of seed stage or very early stage companies—the AgTech Accelerator in Research Triangle Park, NC, and Alexandria LaunchLabs in New York City. Both are accompanied by separate new investment funds of between $10 million and $25 million. Alexandria already has its own venture arm, Alexandria Venture Investments, and is a co-founder of Accelerator Corp., a roughly $60 million early stage VC fund (more on the new funds and Accelerator below). Meanwhile, the company has landed a major deal to build Uber’s headquarters in San Francisco’s Mission Bay area—one of several projects ongoing outside its traditional focus on life sciences. Amidst such moves, its stock hit an all-time high of $114.62 per share on Sept. 8, [Disclosure: Alexandria is a business partner of Xconomy—underwriters, sponsors, and partners have no special influence on our editorial operations.]
The company’s presence in key clusters around the U.S. gives Alexandria co-founder and CEO Joel Marcus, who sits on several boards, including that of BIO (the Biotechnology Innovation Organization) and the Foundation for the National Institutes of Health, a unique view of the innovation landscape. I recently spoke with him about his vision for Alexandria, its new programs for startups, key workforce trends (especially those being driven by the millennials), what he sees ahead for the economy, and the need for more leadership in the life sciences arena, among other topics. Following is an edited transcript of our conversation.
Xconomy: Alexandria doesn’t just build buildings. So I thought a good place to start would be your own description of what Alexandria is, and how that might differ from people’s vision of a real estate company?
Joel Marcus: Over time we’ve evolved our business model to focus on four pillars. First, the real estate. Second, is venture capital, which we think is indispensable to the execution of our real estate mission and our preeminent position in the life-science ecosystem. Third, is thought leadership: it’s crucial to convene high-level thought leaders from across the industry, and we’ve built a collaborative meeting platform to discuss global health challenges and drive progress forward. Then, fourth is corporate social responsibility. We focus on corporate philanthropy, and also the area of sustainability.
X: For people that haven’t been to one of your campuses, how do you describe them?
JM: They are, in fact, campuses. They provide collaborative and innovative environments. We provide great fitness centers, amazing food eateries, like Tom Colicchio’s Riverpark at our New York campus. We have the largest urban garden in New York City (shown below), and have similar gardens on many of our campuses. Our campuses also provide great world-class conference facilities, and we also do a lot of hands-on mentoring. In New York, we’ve recently launched LaunchLabs, which is designed for very early- and pre-venture stage entrepreneurs and companies. We’re providing space, mentorship and a seed fund to help their growth. So it ranges. We try to be very much of a full service operation.
Highly amenitized campuses are now at the forefront of leading companies’ desires. They provide a great tool for recruiting and retention, which these days is becoming very important, especially given that in 10 years the millennials, who want to live where they work, will make up three-quarters of the workforce. Being in a one-off building, or being in a half a floor or one floor of a high-rise building that doesn’t have your name on it, that just doesn’t do it anymore.
X: From your position in clusters around the country, you have a unique view of the economy. What do you see?
JM: On a macro basis, we’re seeing a slow growth mode around the world and the U.S. seems to be in the brightest spot. Therefore, there’s a huge influx of capital seeking safety and yields, and our stock has been one of the great beneficiaries of that—coming from a low in the $70s to about $110 today, just since February, which for a REIT is a pretty dramatic move. The Fed wants to ramp up interest rates while the rest of the world is trying … Next Page »Reprints | Share:
Not long after ViaSat (NASDAQ: VSAT) acquired San Diego-based LonoCloud in 2013, former LonoCloud executives Tom Caldwell and Hossein Eslambolchi met for coffee at the Specialty’s Café and Bakery in University City.
Eslambolchi, who had served as a LonoCloud advisor and CEO, told Caldwell he had an idea for a cybersecurity startup that would use analytic software and machine learning technology to monitor the data flowing in and out of networks for anything that looks out of the ordinary. Caldwell said they soon raised $600,000 from angel investors to start what would become CyberFlow Analytics.
And now CyberFlow Analytics is part of Webroot, a private cloud-based cybersecurity provider based in Broomfield, CO, a suburban community between Denver and Boulder.
“This is a good next chapter for the [CyberFlow] product,” Caldwell said. Financial terms were not disclosed. In the three years since it was founded, CyberFlow raised at least $4 million from investors that include Toshiba America Electronic Components, Siemens Venture Capital, and angel investors.
In a statement today, Webroot said it has acquired CyberFlow’s assets, enhancing its “ability to address the explosion of Internet-connected devices and an increasingly complex threat landscape.” The buyout also fits neatly with the strategy for differentiating itself through “radically different” products that Webroot CEO Dick Williams outlined for Xconomy several years ago.
Caldwell said Webroot has “a good healthy revenue stream, and they’re profitable.” Webroot also plans to expand in San Diego, and Caldwell said he plans to stay on. (Eslambolchi, a former AT&T chief technology officer, was serving as CyberFlow’s executive chairman.)
After acquiring San Diego-based BrightCloud in 2010, Webroot has grown to about 60 employees in San Diego, where work is focused mostly on engineering and product development.
Webroot recently moved into new office space in the University City area, and plans to add most of CyberFlow’s 15 employees, Webroot’s Chad Bacher said last week. The company has over 500 employees in offices around the world, said Bacher, who is Webroot’s senior vice president for product strategy and technical alliances.
San Diego represents a major growth area for Webroot. “This is really the technology hub for our machine learning and threat intelligence services,” Bacher said.
“CyberFlow is really good at finding things that don’t look right, but what we didn’t have was [broader] context. And Webroot’s threat intelligence service provides that context,” said CyberFlow CEO Steve Nye.
The San Diego office is going to become one of Webroot’s centers of analytic software, Nye added. “For us, it’s a great opportunity to make a bigger mark in town besides cybersecurity. This is ‘leaning forward’ cybersecurity.”
Asked to explain, Nye said, “The machine learning and advanced analytics here is very forward leaning. It enables us to move toward more of an AI-based, next-generation cybersecurity.”Reprints | Share:
Across the nation, and even looking back into the past, health was in the headlines this week. Our presidential candidates were either recovering from pneumonia or talking up their health records. New documents came to light that showed the sugar industry paid for studies downplaying the ties between sugar and heart disease. And the drug-price frenzy continued, with Senators Tammy Baldwin and John McCain introducing a new bill to combat price hikes and Mylan CEO Heather Bresch set to testify on Capitol Hill about the EpiPen price increases next week.
Meanwhile, there was yet another twist in the ongoing Duchenne muscular dystrophy drama, more biotech buyouts, new startups, and the filing of a long anticipated IPO. Let’s get right to it.
—Going for IPO: CRISPR Therapeutics, split between Switzerland and Cambridge, MA, filed paperwork to become the third developer of CRISPR-Cas9 gene editing drugs to go public on the Nasdaq. The other two, Editas Medicine (NASDAQ: EDIT) and Intellia Therapeutics (NASDAQ: NTLA), completed IPOs earlier this year… Also in Switzerland, Alzheimer’s drug developer AC Immune outlined plans for a Nasdaq IPO.
—Nine-digit M&A: Horizon Pharma (NASDAQ: HZNP) agreed to buy Novato, CA-based Raptor Pharmaceuticals (NASDAQ: RPTP) for $800 million, or $9 a share. Raptor has two marketed drugs, one to treat a rare metabolic disease and the other to treat lung infections in cystic fibrosis patients… Allergan (NYSE: AGN) said it would buy Vitae Pharmaceuticals (NASDAQ: VTAE) and its pipeline of autoimmune disease drugs, for $639 million, or $21 a share.
—Summit, NJ-based Celgene (NASDAQ: CELG) said this week that its Crohn’s disease drug, mongersen—a key part of Celgene’s inflammatory disease strategy—succeeded in a mid-stage trial. Celgene provided few specifics, however, which will put the spotlight on the data’s full disclosure at a medical meeting later this year…Separately, a notice on the Federal Trade Commission’s website indicated that Celgene may be on the verge of a deal with privately held Swiss company EngMab, which is developing antibody drugs for multiple myeloma.
—French health giant Sanofi (NYSE: SNY) and life-science group Verily, part of Google’s parent Alphabet (NASDAQ: GOOG), unveiled a jointly owned venture called Onduo that will develop diabetes treatments.
—Bezinga first reported this week that Ron Farkas, head of the FDA team that reviewed the Duchenne muscular dystrophy drug eteplirsen from Sarepta Therapeutics (NASDAQ: SRPT), recently left the agency. Sarepta shares climbed more than 20 percent, as investors took the news as a good sign regarding eteplirsen’s chances of approval. The drug is currently under FDA review.
—Shares of Bedminster, NJ, and Irvine, CA-based Aerie Pharmaceuticals (NASDAQ: AERI) climbed more than 60 percent after the company said its combination glaucoma drug, netarsudil/latanoprost (Roclatan), succeeded in a Phase 3 trial. A few doctors expressed concern about the eye redness the drug is known to cause, and its potential impact on patient compliance. Aerie took advantage of the stock bump by filing to raise $50 million.
—Cash For Cambridge Startups: Relay Therapeutics debuted with $57 million from Third Rock Ventures and D.E. Shaw Research. Relay will use Shaw’s supercomputers and other cutting-edge lab tools to explore the shifting shapes of proteins implicated in cancer and other diseases… Sofinnova Partners and Atlas Venture co-led a $35 million Series A round for Delinia, which is developing drugs for autoimmune diseases.
—More Financings: Hayward, CA, and Boston-based Intarcia Therapeutics added $215 million more as it prepares to ask for FDA approval of its implantable diabetes treatment… Riding news that its partner Celgene will file for FDA approval of one of its cancer drugs, Agios Pharmaceuticals (NASDAQ: AGIO) raised $150 million in a stock offering… Dallas-based Peloton Therapeutics, which is developing small molecule cancer drugs, brought in new investor Foresite Capital Management as part of a $52.4 million Series D round…Medford, MA-based Sofregen Medical, which aims to use silk fibers to repair soft tissue defects, raised $6.2 million from Polaris Partners and other unnamed investors.
—Xconomy’s Jeff Engel profiled MIT professor Sangeeta Bhatia and her advocacy for gender diversity in science, engineering, and biotech.
—This year’s prestigious Lasker Awards for biomedical research went to seven scientists for their work on the way cells adapt to fluctuating oxygen supply; hepatitis C biology and drug development; and DNA replication.
—STAT took an in-depth look at employee turnover and morale at the messenger RNA startup Moderna Therapeutics in Cambridge.
—Japanese pharma Eisai launched a research group in Andover, MA, with 90 scientists developing drugs for cancer, dementia, autoimmune diseases, and other disorders.
Alex Lash contributed to this reportReprints | Share:
A cybersecurity report by Ponemon Institute, in association with Keeper Security, found that in the 12 months leading up to June 2016, 55 percent of small and medium-sized businesses (SMBs) experienced a cyber attack, while 50 percent encountered data breaches involving customer and employee information.
These statistics belie the common notion that cybercriminals attack only big businesses. In truth, SMBs and startups are often easier targets, as their defenses tend to be weaker. Limited financial resources make it challenging for these companies to invest in sophisticated security mechanisms or full-fledged IT departments – a fact that hackers and cyber attackers use to their full advantage.
Today, all it takes is one security breach to bring down a company’s brand and reputation. For startups, who depend so much on word-of-mouth recommendations, the impact of a breach could be fatal. Despite this risk, many startups continue to be woefully underprepared. According to the Ponemon Institute survey mentioned earlier, only 14 percent of SMBs rate their ability to mitigate cyber risks, vulnerabilities, and attacks as highly effective.
At a time when authorities such as the World Economic Forum are citing cyber attacks as one of the top global risks, both in terms of likelihood and impact, startups have an important mandate – to make cybersecurity an integral part of their business strategy.
A Good Cybersecurity Program Matters
Today’s startups are more mobile, hyper-connected, social, and globalized than ever – all of which have resulted in more complex data networks and more security risks. Additionally, with the cloud becoming the de facto choice for product development and deployment, newer and more challenging security threats continue to emerge.
Often, the weakest link in the chain could be a third party with inadequate security controls. The onus is on startups to keep these risks at bay, especially as their business amasses a growing volume of sensitive data such as customer contact numbers, credit card data, and intellectual property. Cybersecurity can no longer be a reaction to a threat that has already occurred. Investors and customers expect companies to do all they can to proactively protect the integrity, privacy, and confidentiality of this data.
Ultimately, strong security measures do more than just prevent risks. They foster customer trust, which is essential in driving growth and customer acquisition. In fact, nearly one-third of the respondents in a recent Cisco survey reported that the primary purpose of cybersecurity is to be a growth enabler, while another 44 percent consider cybersecurity a competitive advantage.
Adding further impetus are an increasing number of regulatory initiatives and guidelines around security, including the Cybersecurity Act of 2015, the Cybersecurity National Action Plan (CNAP), and the EU’s General Data Protection Regulation (GDPR).
What then should startups be doing to comply with these mandates, and keep cyber attacks in check?
Treat Cybersecurity as a Business Issue
Cybersecurity is no longer just a compliance or IT checklist concern, but a broader business priority that needs to be aligned with the company’s strategic goals, risk appetite, and risk management framework. In the absence of a CISO or cybersecurity expert, at least one person in the organization – be it a business analyst or enterprise architect — should take on the role of an information security officer, and be responsible for collaborating with the CEO to define a cybersecurity strategy, identify critical data assets, determine security risks and gaps, and implement appropriate controls. There also needs to be a common architecture that consolidates and rationalizes risk and threat data into a “single source of the truth,” which in turn, enables the business, IT, and security functions to collaboratively mitigate risks on time.
Understand the Risks
Many startups, relying on an established cloud services provider, are lulled into a false sense of security, thinking that the data protection measures of the service provider are all they need. This couldn’t be further from the truth. Startups need to be proactive in understanding the risks associated with the service provider, assessing their level of compliance with industry standards, and ensuring effective governance and control through service level agreements and continuous monitoring. It’s also important to clarify the division of responsibilities between a company and its … Next Page »Reprints | Share:
San Diego’s innovation clusters continued to grow in 2015, as local startups, funding deals, and job growth extended an economic growth spurt that began in 2013. That’s according to a report being released today by Connect, the local nonprofit group focused on technology and entrepreneurship.
The Connect Innovation Report found that 405 software, technology, and life sciences startups were created last year in San Diego County.
While that was down from the 449 local startups created in 2014, it is the third consecutive year that more than 400 startups were formed here—a pace that is well beyond the 301 startups per year average over the previous eight years.
Software startups made up nearly 63 percent (255 companies created) of all new high-tech ventures created in 2015, a trend fueled chiefly by software app development, according to the report. Another 82 startups were focused on biotech, pharmaceuticals, or medical devices—making San Diego the top county in California for the creation of new life sciences companies in 2015, the report says.
The annual study draws data from a variety of public and private sources, including the Kansas City, MO-based Kauffman Foundation, which ranked San Diego ninth on its startup activity index, based on measures of startup density and entrepreneurs in a particular region. San Diego also ranked seventh in the Kauffman Index of Growth Entrepreneurship, a new indicator that measures growth entrepreneurship in all industries.
The report on San Diego’s innovation economy also shows:
—Employment at San Diego’s high-tech and life sciences businesses has grown steadily since the great recession, from a low of 135,510 people in 2010 to 149,440 in 2015—or slightly more than 10 percent growth over five years. Employment in the local innovation sector was more robust during the recession than the rest of the San Diego’s private sector, and there were fewer job losses. Job growth last year was led by expansion in San Diego’s life sciences, information and communications technologies (ICT), and aerospace, navigation, and maritime technologies.
—The average salary in … Next Page »Reprints | Share:
Computational thinking is going to be a defining feature of the future—and it’s an incredibly important thing to be teaching to kids today. There’s always lots of discussion (and concern) about how to teach mathematical thinking to kids. But looking to the future, this pales in comparison to the importance of teaching computational thinking. Yes, there’s a certain amount of mathematical thinking that’s needed in everyday life, and in many careers. But computational thinking is going to be needed everywhere. And doing it well is going to be a key to success in almost all future careers.
Doctors, lawyers, teachers, farmers, whatever. The future of all these professions will be full of computational thinking. Whether it’s sensor-based medicine, computational contracts, education analytics, or computational agriculture—success is going to rely on being able to do computational thinking well.
I’ve noticed an interesting trend. Pick any field X, from archeology to zoology. There either is now a “computational X” or there soon will be. And it’s widely viewed as the future of the field.
So how do we prepare the kids of today for this future? I myself have been involved with computational thinking for nearly 40 years now—building technology for it, applying it in lots of places, studying its basic science—and trying to understand its principles. And by this point I think I have a clear view of what it takes to do computational thinking. So now the question is how to educate kids about it. And I’m excited to say that I think I now have a good answer to that—that’s based on something I’ve spent 30 years building for other purposes: the Wolfram Language. There have been ways to teach the mechanics of low-level programming for a long time, but what’s new and important is that with all the knowledge and automation that we’ve built into the Wolfram Language we’re finally now to the point where we have the technology to be able to directly teach broad computational thinking, even to kids.
I’m personally very committed to the goal of teaching computational thinking—because I believe it’s so crucial to our future. And I’m trying to do everything I can with our technology to support the effort. We’ve had Wolfram|Alpha free on the web for years now. But now we’ve also launched our Wolfram Open Cloud—so that anyone anywhere can start learning computational thinking with the Wolfram Programming Lab, using the Wolfram Language. But this is just the beginning—and as I’ll discuss here, there are many exciting new things that I think are now possible.
What Is Computational Thinking?
But first, let’s try to define what we mean by “computational thinking.” As far as I’m concerned, its intellectual core is about formulating things with enough clarity, and in a systematic enough way, that one can tell a computer how to do them. Mathematical thinking is about formulating things so that one can handle them mathematically, when that’s possible. Computational thinking is a much bigger and broader story, because there are just a lot more things that can be handled computationally.
But how does one “tell a computer” anything? One has to have a language. And the great thing is that today with the Wolfram Language we’re in a position to communicate very directly with computers about things we think about. The Wolfram Language is knowledge based: it knows about things in the world—like cities, or species, or songs, or photos we take—and it knows how to compute with them. And as soon as we have an idea that we can formulate computationally, the point is that the language lets us express it, and then—thanks to 30 years of technology development—lets us as automatically as possible actually execute the idea.
The Wolfram Language is a programming language. So when you write in it, you’re doing programming. But it’s a new kind of programming. It’s programming in which one’s as directly as possible expressing computational thinking—rather than just telling the computer step-by-step what low-level operations it should do. It’s programming where humans—including kids—provide the ideas, then it’s up to the computer and the Wolfram Language to handle the details of how they get executed.
Programming—and programming education—have traditionally been about telling a computer at a low level what to do. But thanks to all the technology we’ve built in the Wolfram Language, one doesn’t have to do that any more. One can express things at a much higher level—so one can concentrate on computational thinking, not mere programming.
The Wolfram Language—particularly in the form of Mathematica—has been widely used in technical research and development around the world for more than a quarter of a century, and endless important inventions and discoveries have been made with it. And all these years we’ve also been progressively filling out my original vision of having an integrated language in which every possible domain of knowledge is built in and automated. And the exciting thing is that now we’ve actually done this across a vast range of areas—enough to support all kinds of computational thinking, for example across all the fields traditionally taught in schools.
Seven years ago we released Wolfram|Alpha—which kids (and many others) use all the time to answer questions. Wolfram|Alpha takes plain English input, and then uses sophisticated computation from the Wolfram Language to automatically generate pages of results. I think Wolfram|Alpha is a spectacular illustration—for kids and others—of what’s possible with knowledge-based computation in the Wolfram Language. But it’s only intended for quick “drive by” questions that can be expressed in fairly few words, or maybe a bit of notation.
So what about more complicated questions and other things? Plain English doesn’t work well for these. To get enough precision to be able to get definite results one would end up with something like very elaborate and incomprehensible legalese. But the good news is that there’s an alternative: the Wolfram Language—which is built specifically to make it easy to express complex things, yet is always precise and definite.
It doesn’t take any skill to use Wolfram|Alpha. But if one wants to go further in taking advantage of what computation makes possible, one has to learn more about how to formulate and structure what one wants. Or, in other words, one needs to learn to do computational thinking. And the great thing is that the Wolfram Language finally provides the language in which one can do that—because, through all the work we’ve put into it, it’s managed to transcend mere programming, and as directly as possible support computational thinking.
[Editor’s note: this article is excerpted from a longer blog post, which continues with examples and further context.]Reprints | Share:
Autoimmune diseases occur when our immune systems mistakenly start fighting parts of our own bodies, rather than foreign invaders. The trick is to stop the attack without weakening our body’s defenses. That’s the idea behind a new startup that launched this morning called Delinia.
The company, with operations in Cambridge, MA, and San Francisco, CA, has raised a $35 million Series A round co-led by Sofinnova Partners and Atlas Venture. Delinia is currently led by Saurabh Saha, a venture partner at Atlas, and plans to treat “serious and life-threatening autoimmune diseases,” according to a company statement, though it hasn’t said which ones as of yet.
There are many autoimmune disorders, from rheumatoid arthritis to inflammatory bowel disease and lupus. The common theme is a haywire inflammatory response that causes damage to one part of the body or another—in RA, our joints, and in IBD, our guts. Typical therapies for autoimmune disorders suppress the activity of the immune system. Adalimumab (Humira), for example, is a biologic drug approved for a slew of autoimmune disorders, and it works by tamping down the immune response in diseases like RA or Crohn’s disease (a form of IBD)—but at the cost of leaving the body open to potentially serious infections. Drugs like adalimumab also don’t work for everyone.
Delinia, by comparison, says it aims to beat back the inflammatory response in autoimmune diseases without broadly suppressing the body’s defenses. To do so, it aims to use protein drugs that target regulatory T cells, or Tregs—a type of immune cell that helps regulate the body’s immune response. The goal is to change the levels of Tregs in the body and restore “balance” between various types of immune cells, which in turn supposedly will keep the body’s defenses from commencing a wayward attack.
Delinia’s protein drugs are based on the work of co-founder and chief scientific officer Jeffrey Greve, the former vice president of research at another company developing protein drugs for autoimmune diseases, San Diego’s aTyr Pharma. The $35 million round will get Delinia through a proof-of-concept study for its lead program.
Saha was previously the chief medical officer of Synlogic, another Atlas startup, and president and CSO of BioMed Valley Discoveries before that.
Atlas, meanwhile, is fresh off a win with another autoimmune disease startup. The firm formed Padlock Therapeutics in 2014, a company developing treatments for a variety of autoimmune disorders by targeting so-called PAD enzymes, and sold it to Bristol-Myers Squibb for up to $600 million this past March.Reprints | Share:
MedCrypt, a healthtech cybersecurity startup founded near San Diego, has raised $750,000 in a seed financing round led by Safeguard Scientifics (NYSE: SFE), a private equity fund in suburban Philadelphia that invests in healthcare, fintech, and digital media. (Safeguard Scientifics also is an investor in Sotera Wireless, the San Diego-based maker of wireless devices for remote patient monitoring.)
Various angel investors joined in the deal, MedCrypt co-founder and CEO Mike Kijewski told me in a recent interview.
Founded in January, MedCrypt has been developing cybersecurity technology for healthcare devices that connect to the Internet of Things (IoT), including implantable medical devices and other equipment that has been shown to be vulnerable to hackers. The startup plans to use the capital infusion to refine its prototype security software as a service, and to conduct a pilot project to show that its technology works with medical devices, Kijewski said.
MedCrypt is focused on preventing the sort of thing that happened to St. Jude Medical (NYSE: STJ), Kijewski said. The St. Paul, MN-based medical device maker became the subject of a stock-shorting gambit last month over purported security vulnerabilities in its wireless pacemakers and defibrillators.
A San Francisco investment firm, Muddy Waters Capital, announced on Sept. 25 that it had uncovered “troubling cybersecurity flaws” in St. Jude’s cardiac devices, based on tests conducted by MedSec, a cybersecurity firm. The questions raised by Muddy Waters and MedSec come at a time when St. Jude’s is being acquired by Abbott Laboratories in a deal valued at $25 billion. St. Jude’s has denied that its devices are vulnerable to hacking, and Abbott has said it plans to continue pursuing the deal.
Last week, St. Jude’s filed a lawsuit against Muddy Waters, MedSec, and others—alleging they had intentionally made false and misleading claims about its heart devices to profit from a drop in St. Jude’s stock price.
Whether or not the cardiac devices prove to be hackable, Kijewski said, “What this situation has shown is that a malicious actor does not actually have to exploit a vulnerability to take advantage of it.”
The incident also has spurred questions about the ethics of publicizing the vulnerabilities of implantable medical devices, or for that matter, any connected health technologies. However that debate comes out on Wall Street, Kijewski said it is not exactly in patients’ best interest to disclose how their health data can be hacked.
MedCrypt’s security approach is twofold, Kijewski said. The startup’s system requires multi-factor authentication to access the operating system software that a company uses to manage its healthtech devices; and it encrypts patient data on each device. In a statement released today, MedCrypt says its software allows manufacturers to authenticate users, encrypt data, and cryptographically sign settings and patient prescriptions.
“We don’t need to make devices unhackable,” Kijewski said. “If you spend $20 million to make it unhackable, somebody else will spend $21 million just to get in. Our approach is that we want to make it financially impractical to hack a device. The fact of the matter is that medical devices need better [cybersecurity] solutions.”
What the St. Jude’s case highlights, Kijewski said, is an example of a company “suffering financially because of these security vulnerabilities—without even suffering a breach of their security.”
Kijewski said he began laying the groundwork for MedCrypt with co-founder and CTO Eric Pancoast in 2014, after they sold their medical physics-related software company, Gamma Basics to Varian Medical Systems. The co-founders have been working out of a co-working space in Encinitas, CA, about 30 miles north of San Diego.
Kijewski and Pancoast first teamed up in 2008 while Kijewski was a student at the University of Pennsylvania’s Wharton School. A third MedCrypt co-founder, Penn cryptographer and research professor Brett Hemenway, serves as the company’s chief scientific officer.Reprints | Share:
Recently, the student accelerator program mystartupXX, a joint program of UC San Diego’s Rady School of Management and the Jacobs School of Engineering, won a $50,000 grant from the U.S. Small Business Administration as a Growth Accelerator Fund Competition winner. We were one of 68 winners announced Aug. 31 in the nationwide contest.
This was the third year in a row that mystartupXX has received this award.
As the program director, I was thrilled and honored. It also got me thinking about what was important for winning this competition. I realized that other organizations could benefit from what I’ve learned in this process. So here are a few tips I’d like to share:
First, to win an award, you must be willing to participate and apply. The mystartupXX accelerator is focused on providing mentorship, education, and funding to female-led technology-based startups at UC San Diego. The Growth Accelerator Fund competition is for organizations that help startups grow, become commercially viable, and have an economic impact. When I came across this competition, it seemed like our program could be eligible, so I went ahead and applied. Taking that chance was a great decision for the program—we were able to expand and grow because of the award.
Next, demonstrate your success. When the mystartupXX accelerator started in 2012, we had a very small class of startups in the program. One student in that first group, Rady alum Ashley Van Zeeland, launched the life science startup Cypher Genomics in the accelerator and later developed it into a very successful business. Last year, the company was acquired by Human Longevity, where she is currently the chief technology officer. Her success, and the success of the other startups in the program, garnered attention and interest about the program.
We received more applications to be part of the accelerator and in turn, more successful startups were developed in mystartupXX. Now, the mystartupXX accelerator has supported 26 female-led startups, which have raised more than $8 million in funding. The subsequent times we applied for the SBA award, we were able to show how the program had grown and that it is on a successful trajectory. I believe this was instrumental in repeatedly getting the SBA award.
Finally, don’t be intimidated if your program is new and does not have an extensive track record, or if participating in competitions is not your specialty. When we applied for the first time three years ago, we had just graduated the first cohort of participants and we were still trying to find the most useful program content. Yet, I was willing to apply for the SBA competition because I believe deeply in the work we were doing at mystartupXX. It has been wonderful to play a role in helping female-led startups flourish. As the program has grown, we’ve developed a strong track record, connected to many mentors who worked with the startup companies and the local startup community.
Because I took a few steps out of my comfort zone and applied for the SBA award, the mystartupXX accelerator has grown exponentially. Taking those first steps may not always be easy, but participating in competitions is well worth the time investment.Reprints | Share:
It’s been almost two years since Organovo (NYSE: ONVO) created a new business—using its bio-printing technology to create 3-D samples of living human liver tissue for use in testing the toxicity of pre-clinical drug candidates.
Since then, the San Diego company has built a thriving business as a contract research organization (CRO) that tests experimental drug compounds on bio-printed liver tissue. Each pea-sized sample in a 24-well tray functions (for a week or so) much like a liver in the human body, with cell-to-cell interactions occurring in a 3-D matrix.
According to Organovo CEO Keith Murphy, the technology has been validated across a broad set of safety data, and seven of the top 25 global pharmaceuticals are now customers—including Merck, Roche, Astellas, and Bristol-Myers Squibb. As Murphy put it recently, “We don’t think of ourselves as a bio-printing company any more. We look at ourselves as a human tissues company…We’ll test your compound in our lab as a service.”
Now Organovo is expanding its capabilities. The company announced this week that it has developed a similar testing service for screening pre-clinical drug candidates on living human kidney tissue. Organovo said its bio-printed human kidney tissue simulates the proximal tubule, a key part of the millions of filtering units that make up a kidney—and the primary site of renal toxicity.
The company said it has signed multiple commercial orders for its kidney drug-testing service. Organovo said it also is collaborating on scientific studies of its kidney tissue technology as part of a continuing early access program that includes two big pharma companies.
“We now have a technical capability that no CRO has,” Murphy said. “Think about how compelling it is to change the animal model testing paradigm. The potential of this is to do way better [drug] discovery.”
According to Murphy, the core value of Organovo’s technology is that its 3-D human tissue culture is a much closer approximation to tissue that scientists call in vivo-–within the body—than any conventional tissue used in drug-screening studies. Its 3-D sample facilitates cell-to-cell interactions that are more lifelike than conventional 2-D tissue samples.
With its liver model, for example, Organovo showed that its 3-D liver sample is more sensitive to the toxicity of compounds that were previously deemed to be safe in a standard battery of pre-clinical animal studies and toxicity tests.
Each bio-printed sample of kidney tissue is derived from a cadaver or donor cells, and consists of about 1 million cells. In the wells of a laboratory culture tray, each sample is opaque white and about 3 millimeters (1/8th of an inch) by a half-millimeter.
In March, Organovo CSO Sharon Presnell and other scientists presented data and findings that showed that the company’s “ExVive” Human Kidney samples can carry out a variety of renal and biological functions that are difficult to replicate in a lab sample. Organovo and others made additional presentations this week at the Eurotox 2016 scientific conference in Seville, Spain.
For example, Organovo said its bio-printed kidney tissue demonstrated “functional stability” by continuing to transport the antioxidant glutathione across the cellular membrane of its kidney cells for more than four weeks. Organovo said its kidney samples also react like a real kidney to injury caused by cisplatin, an anti-cancer drug that is a known kidney toxicant.
Murphy and other Organovo executives said the presentations drew a standing-room-only crowd of toxicologists and pharmaceutical industry scientists, and proved to be a seminal event for the company. “It was a turning point because it wasn’t just us talking about our technology,” Murphy said. “It was our customers talking about our technology.”
Murphy added, “With this data, we’re seeing an inflection point in terms of increased customer adoption.”
In a statement, the company estimates that its human kidney tissue service could generate tens of millions of dollars in annual revenue, and represents as much as $100 million in potential future revenue.Reprints | Share:
[Corrected, 9/12/16, 4:28 p.m. See below.] What do cancer experts want? The moon, perhaps. As part of the Obama administration’s “cancer moonshot” program, a panel of scientists this week presented a set of broad, ambitious recommendations to speed up the pace of cancer research, treatment, and prevention.
The scientific goals, writ large, included nationwide data sharing among researchers and drug developers; better prevention through medicine (such as the HPV vaccine) and public education (anti-smoking campaigns); and a registry that could match cancer patients to trials based on genetics and other factors. (The full report is here; a summary is here.)
Xconomy reached Tyler Jacks, the co-chair of the panel and director of the Koch Institute for Integrative Cancer Research at MIT, to ask about the report. A condensed version of our conversation is here.
There were plenty of other cancer headlines, including an Xconomy report about problems that the predominance of immunotherapy—“taking over the field of cancer,” as Jacks put it—is causing for doctors and patients. For more on that and the rest of the week’s news, fire up the roundup rockets. Three… two… one…
—While the moonshot panel painted big pictures, the FDA came out with cancer recommendations of its own. The agency advised women and their doctors not to use screening tests that claim to detect ovarian cancer. They measure the level of a protein, CA-125, in the blood, but they’re not accurate enough, the FDA said, and could prompt women without cancer to pursue more medical procedures, or women with cancer to feel a false sense of security.
—One cancer screening test got positive news from a federal agency. Veracyte (NASDAQ: VCYT) reported that the largest of the eight Medicare administrators has given a tentative thumbs-up to covering Percepta, Veracyte’s test to help determine the likelihood that a suspicious lung nodule is cancerous. Such coverage is critical for a diagnostics business. Last year, Xconomy examined Veracyte and the push to provide tests that rule out cancer. [Updated with the correct number of Medicare administrators.]
—Xconomy reported that doctors are worried patients are signing up for immunotherapy trials but dropping out if they don’t get the drug they seek. The field is also wrestling with growing evidence that immunotherapy drugs actually make tumors look bigger just after treatment, which throws a wrench into traditional methods of assessing whether a drug is working.
—Allergan CEO Brent Saunders criticized “egregious price takers” and pledged his own firm would practice responsible pricing to get out in front of potential legislative action that Democratic presidential candidate Hillary Clinton has been threatening.
—Allergan (NYSE: AGN) also closed a deal, paying $60 million up front to acquire Ann Arbor, MI, gene therapy startup Retrosense Therapeutics, which is developing a potential treatment for retinitis pigmentosa.
—M&A rumors swirled around GW Pharma (NASDAQ: GWPH)… Gilead Sciences (NASDAQ: GILD) officials brushed away the possibility of breaking up… And one big-ticket deal actually went down: Danaher (NYSE: DHR) of Washington, DC, bought diagnostics maker Cepheid (NASDAQ: CPHD) for roughly $4 billion.
—Cambridge, MA-based Agios Pharmaceuticals (NASDAQ: AGIO) said this week that development partner Celgene (NASDAQ: CELG) aims to file for accelerated approval of one of Agios’s blood cancer drugs, enasidenib, by the end of the year. The application could put Agios well ahead of typical cancer drug development timelines; shares spiked more than 20 percent on the news.
—Bay Area regulatory blues: Biomarin Pharmaceutical (NASDAQ: BMRN) of San Rafael, CA, had its FDA review of cerliponase alfa (Brineura) delayed. The agency will now decide on approval by April 27 of next year… Dynavax Technologies (NASDAQ: DVAX) of Berkeley, CA, said the FDA’s advisory committee scheduled to weigh in on the company’s hepatitis B vaccine decided to cancel the meeting. Investors knocked about 30 percent off Dynavax’s share price last Friday, with only a modest recovery this week.
—Bluebird Bio (NASDAQ: BLUE) started a late stage trial of its gene therapy, LentiGlobin, in patients with the blood disease beta-thalassemia. Bluebird is tinkering with LentiGlobin to boost its effectiveness, and is also excluding a subgroup of patients—those with two copies of a genetic mutation called b0—who haven’t responded to LentiGlobin in the past. Bluebird said the FDA would let it incorporate the changes into the study, rather than start a new set of trials.
—Seattle’s Juno Therapeutics (NASDAQ: JUNO) published positive data from a study of one of its T cell treatments, JCAR014, in 32 people with advanced non-Hodgkins lymphoma. The treatment for one group of the patients included a cell-boosting chemotherapy, fludarabine, that Juno said was responsible for three deaths earlier this year in a trial for the product JCAR015. Juno briefly halted then restarted that trial without fludarabine, but explained that the chemo would remain part of the drug regimen in other trials and experimental products.
—More clinical news: Shares of CoLucid Pharmaceuticals (NASDAQ: CLCD) more than doubled after the company said its experimental migraine drug, lasmiditan, hit all goals in the first of two Phase 3 trials. Results from the … Next Page »Reprints | Share:
The Obama administration wants to speed up cancer research with its “Cancer Moonshot” program—packing 10 years’ worth of work on prevention and treatment programs into five is one of its rallying cries. Agreeing on what to do, and how to do it, is the first step. The FDA agreed this summer to reorganize the way it evaluates drugs, diagnostics, and other cancer-related products.
This week, a panel of 28 scientists presented the National Cancer Institute’s National Cancer Advisory Board a set of recommendations and national priorities for basic research, clinical trial practice, and drug development. In an interview with Xconomy, panel co-chair Tyler Jacks acknowledged the recommendations were broad, with many details yet to be worked out. (So broad, in fact, that they elicited no small amount of Internet eye-rolling, including this tweet Thursday from David Shaywitz, CMO of genomic data firm DNANexus.)
Moonshot recommendations remind me of Bart Giamatti's famous edict on day he assumed Presidency of Yale… pic.twitter.com/ZU3ZxJsVCQ
— David Shaywitz (@DShaywitz) September 9, 2016
A set of recommendations for policies and reimbursement is forthcoming from a different moonshot group.
The scientific goals, writ large, included nationwide data sharing among researchers and health practitioners; better prevention through medicine (such as the HPV vaccine) and public education (anti-smoking campaigns); and a registry that could match cancer patients to trials based on genetics and other factors. (The full report is here; a summary is here.)
Xconomy reached Jacks, who is director of the Koch Institute for Integrative Cancer Research at MIT, after the panel made its presentation in Washington, DC, this week. The following is an edited, condensed version of our conversation.
X: The report starts by recommending a national cancer patient network. Is this akin to a nationwide study, like the government’s precision medicine initiative?
TJ: No, it’s not a national study or clinical trial. It’s characterization of individuals’ cancers, a way to apply analysis to patients all over the country. Included is the idea that when a patients signs up they’ll get better information about their own cancer—bringing the science to the patient—and better access to clinical trials. If you analyze the genome of a patient’s tumor, a mutation in gene X might make [the tumor] sensitive to drug Y. Once they’re in the system, you can get them onto a trial. It’s related to the MATCH trial being run by the National Cancer Institute.
X: It seems every day there are more examples of one mutation identified as the driver of a cancer. But is there a danger of relying too heavily just on genomics to determine the course of a patient’s treatment?
TJ: We know that existence of particular mutations can predict sensitivity to some drugs, but in some cases they don’t. And in many patients there are mutations without appropriate drugs. We’re going to need other biomarkers to indicate different treatment strategies, such as RNA expression or protein expression. Right now, getting the genomic profile is easiest. But at the end of the day [the network] will integrate other data types.
X: This sounds a lot like the GENIE project to pool patient data among seven top cancer centers.
TJ: Yes. This is another [initiative] to create systems to more effectively catalog the collection of mutations that exist. GENIE is one of several ongoing. The hope is that we can bring it to a broader array of patients.
X: Even among the GENIE members, there are varying levels of resources to build the data infrastructure, convert existing data, and pay for things like genomic tests. How will you implement this across the country?
TJ: Important new infrastructures will have to be built, for sure. There are policy issues. And who will pay for the genotyping of every patient?
X: You don’t think that sequencing prices will come down enough? … Next Page »Reprints | Share:
Throughout history, innovative humans have developed technology to overcome or adapt to challenges in the natural world. As it turns out, some of our most important advances were actually inspired by a naturally occurring group of materials: polymers.
Derived from Greek words meaning “many parts,” the word polymer refers to a large molecule composed of multiple repeating units. This structure makes polymers strong, but lightweight, and flexible—properties that come in handy for a wide range of purposes.
Unknown to most people, natural polymers include cotton, wool, leather, and silk, which have been used to make clothing for thousands of years. Silk, produced naturally by insects, was even deployed in military conflicts. Lightweight, but impenetrable to arrows, woven silk undergarments helped Mongol invaders sweep across Europe and defeat knights wearing heavier metal armor as far back as the 13th century. During the U.S. Civil War, clothing makers added cellulose—a building block of plant cells—to military uniforms.
As scientists studied polymers, they found different ways to use or improve them. For example, experiments demonstrated that adding sulphur to cellulose, a process known as vulcanization, created a polymer that was less sticky, enabling production of rubber hoses, gloves, belts, and more.
Those successes helped inspire a multitude of manmade polymers that we know today, including rayon, nylon, plastics, and Teflon. Kevlar, developed 50 years ago, is lightweight and flexible, but super-strong. Today, you can find it in products ranging from bicycle tires to mooring lines and body armor.
Other polymer-based products make our lives easier and safer. They reduce energy costs and enable medical advances. Some examples include shatterproof windshields, lightweight composite materials in airplanes, and stitches that gradually dissolve after they are applied to a wound.
Researchers continue to discover new, powerful ways to use polymers. In fact, the 2000 Nobel Prize for chemistry was won by a team of scientists who discovered that polymers can be modified to conduct electricity. This could support innovative products ranging from organic solar cells and light-emitting diodes to next-generation coatings on stealth aircraft. Conductivity could also enable smart textiles, which would mimic human skin by reacting to electrical signals like temperature change. Printing miniature electric circuits on clothing could allow it to monitor the wearer’s vital signs, and even call for help in the event of an emergency.
And at the University of Illinois at Urbana-Champaign, scientists have synthesized a self-healing polymer, which might one day make spacecraft less reliant on human repairs. Imagine a coat of paint on a house that repairs its own cracks, or having your favorite shirt mend itself after snagging on a nail. Self-healing polymers might even make buildings more resilient, better able to withstand earthquake damage.
It’s also important to consider the environmental impact from polymer products. For example, most plastics are not biodegradable, and since we currently recycle only a fraction of what we create, too much ends up in landfills or in the natural environment. Going forward, we’ll need to improve our disposal and recycling performance and consider new technologies that accelerate the biodegradation of obsolete materials.
Ultimately, polymers have reduced our dependence on scarce natural resources, including wood from trees and metals which must be mined and processed. And the reduced weight of polymer-based containers compared to glass and metals helps conserve fuel used for transportation, reducing pollution.
The benefits of polymers are enormous and many are still being discovered. Continuing research will deliver innovations that improve our lives both in the near future and for many years to come.Reprints | Share:
Retrophin, the drug developer founded by controversial former biotech executive Martin Shkreli, has posted positive results from a midstage study for its treatment for a rare kidney disease.
San Diego-based Retrophin said that its drug, sparsentan, outperformed another drug sometimes used to manage a rare kidney disease, called focal segmental glomerulosclerosis (FSGS), in a Phase 2 clinical trial. The kidney disorder doesn’t have any FDA-approved therapies, Retrophin says, but several drugs are currently used off-label to treat it, including steroids, immune system suppressants, and diuretics.
Patients who received sparsentan had a 44.8 percent reduction in a symptom of the disease that’s associated with a breakdown of normal kidney function, according to the initial trial results. Meanwhile, patients who received irbesartan—one of a few different drugs used to manage FSGS—saw the symptom decline by 18.5 percent.
Retrophin (NASDAQ: RTRX) shares rose $4.96 cents by midday Wednesday, up 30.3 percent to $21.25 on the Nasdaq. The company plans to present detailed results at an upcoming medical meeting or in a peer-reviewed journal, it said in a statement.
The results exceeded expectations, according to a note from analysts at Leerink Partners, who said the drug has a 75 percent chance to earn FDA approval. The analysts said they were cautiously optimistic that Retrophin will meet the next milestone: earning an accelerated approval process from the FDA for the drug. Leerink expects the company to meet with the FDA later this year.
Retrophin’s involvement with sparsentan began with Shkreli, the company’s former CEO, who has since been characterized as an archetype of pharmaceutical industry greed rather than innovation. He licensed the drug from San Diego-based Ligand Pharmaceuticals (NASDAQ: LGND) in March 2012. Months later, Shkreli raised a $4 million Series A round for Retrophin from the hedge fund that he also ran, MSMB Capital.
After in-licensing and developing multiple other compounds and taking the company public through a reverse merger, Shkreli was ousted from Retrophin in late 2014—shortly after he raised the price on an old drug used for treating a condition related to kidney stones, which the company acquired, from $1.50 to $30 apiece. That would prove to be a harbinger of things to come.
After leaving Retrophin, Shkreli founded a new biotech, Turing Pharmaceuticals where he doubled down: He raised the price of another drug, Daraprim, used to treat parasitic infections that often afflict people with HIV. This time, he increased the price from $13.50 to $750 a pill. To say the least, there was widespread public outrage. While the incident undoubtedly made him famous, it also turned him into a global punching bag for price gouging. To boot, he was pushed out of Turing, indicted on fraud charges, and sued by Retrophin.
Even so, acquisitions that Shkreli made in Retrophin’s early days, such as sparsentan, can be partly credited for making Retrophin a potential success. The Leerink analysts placed a $32 per share price target on the stock.
FSGS affects an estimated 40,000 patients in the U.S. and can lead to end-stage renal disease after progressive scarring of the kidney, according to Retrophin. The Phase 2 trial studied sparsentan’s ability to reduce a measurement of a protein in a patient’s urine, known as proteinuria. The symptom is caused by a breakdown of the kidney’s normal filtration mechanism. Reducing levels of proteinuria is associated with a lower risk of FSGS, Retrophin says.
Other symptoms include edema, high cholesterol, and high blood pressure, according to nonprofit NephCure Kidney International.Reprints | Share: